Growth Hormone in Amyotrophic Lateral Sclerosis

Federico II University

Status and phase: Completed; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Growth Hormone (Somatropin); Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT00635960

SLA_GH_1

Details and patient eligibility

About

Several drugs have been proposed for ALS. These drugs included: Topiramate, Lamotrigine, creatine, Vit. E, Pentoxifylline, etc. Although most of the trials showed a positive trend, none of them reached a statistically significant result. The only exception is the Riluzole trial, that demonstrated a small but significant reduction in mortality between treated and untreated patients. Aim of our study is to determine if the add-on of GH to treatment with Riluzole is able to reduce neuronal loss in the motor cortex of ALS patients.

Full description

Several drugs have been proposed for ALS. These drugs included: Topiramate, Lamotrigine, creatine, Vit. E, Pentoxifylline. Although most of the trials showed a positive trend, none of them reached a statistically significant result. The only exception is the Riluzole trial, that demonstrated a small but significant reduction in mortality between treated and untreated patients. When administered to SOD-1 transgenic mice, IGF-I prolongs survival, ameliorates muscular strength, and reduces weight and motor neuron loss, astrocyte gliosis, and ubiquitin positive protein inclusions.

Two clinical trials have been performed in ALS patients with s.c. administration of IGF-I indicating a possible beneficial effect, and a third clinical trial is in progress. Methionyl growth hormone (mGH) showed no effect on survival, disease progression and muscular strength. MGH was administered at a fixed dose and peripheral production of IGF-I appeared to be normal. We propose a double-blind trial of Growth Hormone (GH) as add-on therapy to Riluzole, with an individually regulated dose based on the peripheral response of IGF-I. Aim of our study is to determine if the add-on of GH to treatment with Riluzole is able to reduce neuronal loss in the motor cortex of ALS patients. As secondary objectives, effect of GH on mortality, QoL, and motor function will be assessed.

Enrollment: 40 estimated patients

Sex: All

Ages: 40 to 85 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Definite/probable ALS according to El Escorial criteria
• Aged > 40, < 85 years
• Progression from onset
• Disease duration ≤3 years
• Treatment with Riluzole

Exclusion criteria

• Rapid disease progression in the first 6 months after diagnosis
• Patients with tracheostomy and/or Gastrostomy
• Disease duration > 3 years
• Patient with exclusive bulbar or 2° motorneuron involvement
• Hepatic/renal failure
• Pregnant or breastfeeding
• Signs of active neoplasia
• Complicated Diabetes
• Severe hypertension
• Unable to undergo MRI exams

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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40 participants in 2 patient groups, including a placebo group

1

Experimental group

Description:

Patients randomly assigned to treatment

Treatment:

Drug: Growth Hormone (Somatropin)

2

Placebo Comparator group

Description:

Patients randomly assigned to placebo

Treatment:

Drug: Placebo